WO2014118817A3 - Plaquettes transfectées par un matériau génétique exogène et microparticules plaquettaires obtenues à l'aide des plaquettes transfectées, procédé pour leur préparation et leurs utilisations - Google Patents

Plaquettes transfectées par un matériau génétique exogène et microparticules plaquettaires obtenues à l'aide des plaquettes transfectées, procédé pour leur préparation et leurs utilisations Download PDF

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Publication number
WO2014118817A3
WO2014118817A3 PCT/IT2014/000028 IT2014000028W WO2014118817A3 WO 2014118817 A3 WO2014118817 A3 WO 2014118817A3 IT 2014000028 W IT2014000028 W IT 2014000028W WO 2014118817 A3 WO2014118817 A3 WO 2014118817A3
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WO
WIPO (PCT)
Prior art keywords
transfected
platelets
genetic material
preparation
exogenous genetic
Prior art date
Application number
PCT/IT2014/000028
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English (en)
Other versions
WO2014118817A2 (fr
Inventor
Paolo Gresele
Marco MALVESTITI
Original Assignee
Paolo Gresele
Malvestiti Marco
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Paolo Gresele, Malvestiti Marco filed Critical Paolo Gresele
Priority to EP14717082.3A priority Critical patent/EP2951292B1/fr
Priority to SI201431873T priority patent/SI2951292T1/sl
Priority to DK14717082.3T priority patent/DK2951292T3/da
Priority to US14/764,561 priority patent/US20150361453A1/en
Priority to ES14717082T priority patent/ES2884727T3/es
Priority to PL14717082T priority patent/PL2951292T3/pl
Publication of WO2014118817A2 publication Critical patent/WO2014118817A2/fr
Publication of WO2014118817A3 publication Critical patent/WO2014118817A3/fr
Priority to US17/814,845 priority patent/US20230048730A1/en
Priority to US17/814,860 priority patent/US20220411823A1/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/19—Platelets; Megacaryocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0644—Platelets; Megakaryocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Manufacturing & Machinery (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La présente invention concerne des plaquettes transfectées avec un matériau génétique exogène et des microparticules dérivées desdites plaquettes transfectées ayant un pourcentage élevé de transfection et pouvant transporter et transfecter des cellules receveuses avec un matériau génétique, ces plaquettes pouvant ensuite être utilisées par exemple en thérapie cellulaire et génique. L'invention concerne en outre un procédé de préparation de plaquettes matures transfectées avec un matériau génétique exogène et des microparticules dérivées desdites plaquettes transfectées ainsi que des microparticules dérivées de plaquettes matures transfectées, ledit procédé permettant d'obtenir des pourcentages de transfection élevés.
PCT/IT2014/000028 2013-01-30 2014-01-30 Plaquettes transfectées par un matériau génétique exogène et microparticules plaquettaires obtenues à l'aide des plaquettes transfectées, procédé pour leur préparation et leurs utilisations WO2014118817A2 (fr)

Priority Applications (8)

Application Number Priority Date Filing Date Title
EP14717082.3A EP2951292B1 (fr) 2013-01-30 2014-01-30 Plaquettes transfectées par un matériau génétique exogène et microparticules plaquettaires obtenues à l'aide des plaquettes transfectées, procédé pour leur préparation et leurs utilisations
SI201431873T SI2951292T1 (sl) 2013-01-30 2014-01-30 Trombociti, transfektirani z eksogenim genetskim materialom, in trombocitni mikrodelci, dobljeni s transfektiranimi trombociti, postopek za pripravo in uporabe le-teh
DK14717082.3T DK2951292T3 (da) 2013-01-30 2014-01-30 Blodplader transficeret af eksogent materiale og blodplademikropartikler opnået med de transficerede blodplader, fremgangsmåde til fremstilling og anvendelser deraf
US14/764,561 US20150361453A1 (en) 2013-01-30 2014-01-30 Platelets transfected by exogenous genetic material and platelet microparticles obtained by said transfected platelets, method for the preparation and uses thereof
ES14717082T ES2884727T3 (es) 2013-01-30 2014-01-30 Plaquetas transfectadas por material genético exógeno y micropartículas plaquetarias obtenidas por dichas plaquetas transfectadas, procedimiento de preparación y usos de las mismas
PL14717082T PL2951292T3 (pl) 2013-01-30 2014-01-30 Płytki krwi transfekowane za pomocą egzogennego materiału genetycznego oraz mikrocząstki płytkowe uzyskane przy użyciu wymienionych transfekowanych płytek krwi, sposób ich wytwarzania i zastosowanie
US17/814,845 US20230048730A1 (en) 2013-01-30 2022-07-26 Platelets transfected by exogenous genetic material and platelet microparticles obtained by said transfected platelets, method for the preparation and uses thereof
US17/814,860 US20220411823A1 (en) 2013-01-30 2022-08-05 Platelets transfected by exogenous genetic material and platelet microparticles obtained by said transfected platelets, method for the preparation and uses thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
ITRM2013A000054 2013-01-30
IT000054A ITRM20130054A1 (it) 2013-01-30 2013-01-30 Piastrine trasfettate con materiale genetico esogeno e microparticelle derivate da dette piastrine trasfettate, metodo per la loro preparazione e loro usi.

Related Child Applications (3)

Application Number Title Priority Date Filing Date
US14/764,561 A-371-Of-International US20150361453A1 (en) 2013-01-30 2014-01-30 Platelets transfected by exogenous genetic material and platelet microparticles obtained by said transfected platelets, method for the preparation and uses thereof
US17/814,845 Division US20230048730A1 (en) 2013-01-30 2022-07-26 Platelets transfected by exogenous genetic material and platelet microparticles obtained by said transfected platelets, method for the preparation and uses thereof
US17/814,860 Division US20220411823A1 (en) 2013-01-30 2022-08-05 Platelets transfected by exogenous genetic material and platelet microparticles obtained by said transfected platelets, method for the preparation and uses thereof

Publications (2)

Publication Number Publication Date
WO2014118817A2 WO2014118817A2 (fr) 2014-08-07
WO2014118817A3 true WO2014118817A3 (fr) 2014-10-09

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IT2014/000028 WO2014118817A2 (fr) 2013-01-30 2014-01-30 Plaquettes transfectées par un matériau génétique exogène et microparticules plaquettaires obtenues à l'aide des plaquettes transfectées, procédé pour leur préparation et leurs utilisations

Country Status (10)

Country Link
US (3) US20150361453A1 (fr)
EP (1) EP2951292B1 (fr)
DK (1) DK2951292T3 (fr)
ES (1) ES2884727T3 (fr)
HU (1) HUE055345T2 (fr)
IT (1) ITRM20130054A1 (fr)
PL (1) PL2951292T3 (fr)
PT (1) PT2951292T (fr)
SI (1) SI2951292T1 (fr)
WO (1) WO2014118817A2 (fr)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020113101A1 (fr) 2018-11-30 2020-06-04 Cellphire, Inc. Plaquettes chargées d'agents anticancéreux
US20210180016A1 (en) * 2019-11-27 2021-06-17 Cellphire, Inc. PLATELETS LOADED WITH mRNA
EP3886879A4 (fr) * 2018-11-30 2022-12-07 Cellphire Inc. Plaquettes en tant qu'agents de livraison
CA3138529A1 (fr) 2019-05-03 2020-11-12 Cellphire, Inc. Materiaux et procedes de production de produits sanguins
US20210308185A1 (en) 2020-02-04 2021-10-07 Cellphire, Inc. Methods of treating acquired hemophilia with anti-fibrinolytic loaded platelets
CA3150936A1 (fr) 2019-08-16 2021-02-25 Cellphire, Inc. Thrombosomes en tant qu'agent desactivateur d'antiagregant plaquettaire
US20210299180A1 (en) * 2020-03-27 2021-09-30 Platelet Biogenesis, Inc. Novel anucleated cells and uses thereof
IT202100021779A1 (it) 2021-08-11 2023-02-11 Plasfer S R L Piastrine trasfettate con sirna e loro usi terapeutici

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Publication number Priority date Publication date Assignee Title
WO2000027795A1 (fr) * 1998-11-12 2000-05-18 Invitrogen Corporation Reactifs de transfection

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS: "Polyethylenimine", XP002727771, Retrieved from the Internet <URL:http://en.wikipedia.org/wiki/Polyethylenimine> [retrieved on 20140724] *
FUJIMOTO T.T. ET AL: "Production of functional platelets by differentiated embryonic stem (ES) cells in vitro", BLOOD, vol. 102, no. 12, 2003, pages 4044 - 4051, XP002695897 *
HONG W. ET AL: "Transfection of human platelets with short interfering RNA", CLIN. TRANS. SCI., vol. 4, 2011, pages 180 - 182, XP002695898 *
KONDKAR ALTAF A ET AL: "Transfection of Human Platelets Down-Regulates Endogenous mRNA", 22 November 2009 (2009-11-22), 51ST ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY, USA, XP002695899, Retrieved from the Internet <URL:https://ash.confex.com/ash/2009/webprogram/Paper22119.html> [retrieved on 50140725] *
OHMORI T ET AL.: "Efficient expression of a transgene in platelets using simian immunodeficiency virus-based vector harboding glycoprotein Ibalpha promoter: in vivo model for platelet-targeting gene therapy", FASEB J, vol. 20, 2006, pages E769 - E779, XP002695896 *

Also Published As

Publication number Publication date
DK2951292T3 (da) 2021-08-23
EP2951292B1 (fr) 2021-05-26
PL2951292T3 (pl) 2021-12-13
EP2951292A2 (fr) 2015-12-09
ES2884727T3 (es) 2021-12-10
ITRM20130054A1 (it) 2014-07-31
WO2014118817A2 (fr) 2014-08-07
US20220411823A1 (en) 2022-12-29
US20230048730A1 (en) 2023-02-16
SI2951292T1 (sl) 2021-11-30
HUE055345T2 (hu) 2021-11-29
US20150361453A1 (en) 2015-12-17
PT2951292T (pt) 2021-08-30

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